New approach has the potential to improve the chances of medicines for rare and orphan diseases receiving positive decisions from NICE and other pricing watchdogs

For the first time, pharmaceutical companies are sponsoring a project, led by Duchenne UK, to generate, align and share disease-level data across an entire condition for health technology assessments (HTA). The collaboration, known as HERCULES, will focus on medicines for Duchenne muscular dystrophy.

The HERCULES industry partners announced today are:

Pfizer Inc

PTC Therapeutics International Ltd

Roche

Sarepta Therapeutics, Inc

Solid Biosciences

Summit Therapeutics Plc

Wave Life Sciences USA, Inc

The unique initiative, launched by the Duchenne muscular dystrophy research charity Duchenne UK, aims to radically simplify the way evidence is generated for submissions to HTA bodies, such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de Santé (HAS). The University of Leicester and University of Sheffield have also partnered on HERCULES in the development of a quality of life metric and data analysis.

While this new initiative focuses on Duchenne muscular dystrophy, it paves the way for similar approaches in other rare diseases, and has the potential to better demonstrate the value of medicines for rare and orphan diseases. It is hoped that greater certainty over the value of these medicines could help to improve their chances of receiving positive HTA decisions.

Rare conditions affect around 3.5 million people in the UK[1] and 30 million people across Europe[2]. Yet only 5 per cent of rare diseases have a licensed treatment option in the UK[3]. Small patient populations and limited resources to build a robust evidence base are key hurdles in developing treatments for rare diseases. These obstacles tend to hinder the HTA approval of potentially life-changing treatments for rare diseases and their subsequent availability to NHS patients, even for medicines that have received regulatory approval.

Emily Crossley, Co-CEO of Duchenne UK, said: “As a parent of a child with Duchenne, it can be heartbreaking to know that access to life-changing treatments might be delayed because the assessment bodies don’t have the right evidence to make a decision. By pooling evidence through HERCULES, all partners involved can avoid ‘reinventing the wheel’ by producing evidence for similar submissions.

“We’re delighted that our industry partners have joined us to lead the way in this collaborative approach. HERCULES has the potential to transform health technology assessment submissions in Duchenne muscular dystrophy and other rare diseases. This could not only help industry and assessment bodies such as a NICE, but ultimately ensure that new treatments for DMD get to the boys who need them the most.”

Fleur Chandler, Future Pipeline Head, VEO at GSK R&D and a member of Duchenne UK’s Patient Advisory Board added: “With HERCULES, we have a patient organisation taking the lead to solve a problem that the industry has talked about for a long time, but where limited progress has been made to date. HERCULES gives us the opportunity to develop a much more robust evidence base for Duchenne muscular dystrophy. Some companies can dedicate considerable time and resource to developing the economic modelling and quality of life measures for their submissions. However, this is not the case for all, which runs the risk of uncertainty in decision making and, ultimately, patients losing out.”

Janis Clayton, General Manager at PTC Therapeutics Ltd, said: “HERCULES, led by Duchenne UK, provides an excellent opportunity for innovation and collaboration, bringing together as it does patient organisations, industry and academia. PTC are very pleased to be supporting, and actively involved in, this appropriately ambitious project.”

Duchenne UK would like to thank CMS Cameron McKenna Nabarro Olswang LLP for their legal support and advice on Project Hercules.

HERCULES will help produce a comprehensive, standardised evidence base for new treatments that have the potential to change the lives of patients with DMD. Led by Duchenne UK with seven pharmaceutical industry sponsors and two academic partners, the project will enable a stronger evidence base for new therapies for Duchenne Muscular Dystrophy (DMD) required by regulatory bodies, such as the National Institute for Health and Care Excellence (NICE). These evidence requirements are very different from the evidence required to grant regulatory approval for a medicine.

Phase One of HERCULES will begin in early 2018, and focus on understanding the current evidence base, the cost of DMD and lives lost to it, as well as current measures of success in DMD clinical trials. This unique project with seven industry sponsors will enable development of the critical components required for a successful HTA submission including:

There are around 2,500 boys affected by DMD in the UK and around 300,000 worldwide. It is classified as a rare disease

DMD is the most common fatal genetic disease diagnosed in childhood, almost exclusively affecting boys

Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. There is no treatment or cure

About Duchenne UK

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for DMD. Duchenne UK has raised more than £5.5million in five years, and spent or committed £5million to DMD research projects – more than any other organisation

Duchenne UK is leading the drive towards the efficient repurposing of existing medicines to bring new treatments for DMD

Duchenne UK believes that every child with Duchenne should be given the opportunity to access a clinical trial. Duchenne UK created the DMD Hub in 2016 to expand and develop the infrastructure for DMD trials in the UK and accelerate access to clinical studies for those who are interested in participating

The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell

Visit duchenneuk.org for more information or follow @DuchenneUK on Twitter.

Summit Therapeutics plc have announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. The data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies.

Related

Summit Therapeutics plc have announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. The data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies.

Yesterday, the new Care Considerations for Duchenne were published. We strongly encourage all parents, caregivers and patients to read these updates to the standards of care to ensure that everyone living with Duchenne has access to the best possible care

We’re pleased to share the latest coverage of Hercules. The launch of the project, with our seven industry partners, has received coverage in 10 publications. HERCULES is collaboration between Duchenne UK, and pharmaceutical companies to increase the chances of patients with DMD of accessing innovative treatments.

Duchenne UK is delighted to have been chosen by Marshall Wace as their charity of the year and to be awarded a donation of $100,000. The donation from Marshall Wace will fund 10 boys with Duchenne on our Tamoxifen Trial.

The DMD-Hub was created by Duchenne UK and leading neuromuscular clinicians. The DMD-Hub is addressing the lack of capacity in the UK for clinical trials in DMD. During the first year of operation the DMD Hub has successfully achieved, even surpassed, the immediate priorities identified through the Newcastle Plan.

We would like to congratulate the Joining Jack team for their recent victory! Joining Jack took a team to the Dubai 7's international rugby competition, and they came back champions! Alex Johnson, founder of Joining Jack and Jacks Mum, tells us more about Joining Jack's victory at the Dubai 7s.

It has been a busy year at Duchenne UK. Our mission is to fund and accelerate treatments and find a cure for Duchenne muscular dystrophy. We look at where we can add value to the drug development pathway, and how we can help to accelerate research that will benefit the whole community.

Today, December 14th, marks the publication of Janet Hoskin's book: 'A Guide to Duchenne Muscular Dystrophy: Information and Advice for Teachers and Parents'. We are offering free copies to families affected by DMD, please contact us to request a copy.

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Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

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